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Charcot-Marie-Tooth Clinical Outcome Measure Update

Professor Joshua Burns has launched an updated web-based tool too assist researchers and clinicians to measure the level of disability in Charcot-Marie-Tooth disease (CMT). This new website enables users to rapidly score clinical outcome measures including: the Charcot-Marie-Tooth disease Pediatric Scale (CMTPedS), CMT Infant Scale (CMTInfs), CMT Functional Outcome Measure (CMT-FOM), CMT Neuropathy Score (CMTNS) […]

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Recruitment Request: Understanding patients’ and carers’ decision making in Spinal Muscular Atrophy Care

Associate Professor Michelle Farrar and colleagues from Sydney Children’s Hospital (Randwick) are conducting a new research project to lean more about what factors are important when making decisions related to treatment for  spinal muscular atrophy (SMA). They are seeking the views of parents/carers of people with SMA, health care providers and the wider community. Please find attached more […]

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Early Access Program for Nusinersen

Early Access Program for Nusinersen   There has been a lot of activity in the clinical trial space for the treatment of Spinal Muscular Atrophy (SMA) over the last two years, and Nusinersen, has been identified as the first effective drug to treat SMA Type 1.   Developed in California, Nusinersen (know in the USA […]

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INMR diagnostic meetings schedule

Please see below for dates of The Institute for Neuroscience and Muscle Research (INMR) at The Children’s Hospital at Westmead diagnostic meetings schedule. Genetic Muscle Disease Diagnostic Meeting: As we move away from tissue diagnosis towards genetic diagnosis, this forum is to discuss difficult cases and to pool thoughts on the interpretation of results (incorporating […]

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